Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes. (2020)


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Bibliographic Information

Digital Object Identifier:

PubMed Identifier: 31919491

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Type: Journal Article/Review

Volume: 29

Parent Publication: Human molecular genetics

Issue: 12

ISSN: 0964-6906